Using Investigational Medicines
What are investigational medicines?
Investigational medicines are used when you are seriously ill but not enrolled in
a clinical trial. These medicines are being studied but have not yet been approved
as safe and effective by the FDA. It is sometimes called compassionate medicine use.
Normally, investigational medicines have strict testing through many stages of clinical
trials before they are available to the public. They are tested for safety and side
effects. They are also tested to see how well they work and at what doses, and with
what conditions or diseases they are to be used. The process may take many years. In
certain cases, these medicines can be used outside of a clinical trial.
Investigational medicine use is legal. The FDA has been approving investigational
medicines to be used in this way for critically ill people since 1987.
Who is eligible to use these medicines?
The most common way people access unapproved medicines is by enrolling in a clinical
trial. This is the safest route. The FDA closely regulates clinical trials, and people
taking new medicines are carefully monitored. Not everyone will qualify for a clinical
trial. Because the use of new, unapproved medicines is risky, only certain people
who are not enrolled in clinical trials can get investigational medicines for compassionate
use. According to guidelines from the National Cancer Institute (NCI), you are eligible
Have used standard treatments and they didn't work
Have an illness that is life-threatening
Are ineligible for any ongoing clinical trials
Have no acceptable treatment options
Have a cancer diagnosis for which an investigational medicine has shown some benefit
and is currently being studied in clinical trials
Are likely to have benefits that outweigh the risks involved
Getting investigational medicines
To use investigational medicines, your healthcare provider has to apply for them through
a treatment investigational new drug application (treatment IND). The application
process includes information on your condition and past treatments.
The most common way to get approval for an investigational medicine outside of clinical
trials is through expanded access or special exception or single-patient exception.
Expanded access uses medicines that have been well-tested and are nearing FDA approval.
The medicine must show in studies that it is effective in treating a specific cancer.
The program is offered by a medicine company, and the medicine is given out to a group
Special exceptions are individual requests (often by a healthcare provider) made to
medicine companies or to whomever is overseeing a program or clinical trial. The decision
to give out the medicine is weighed on a case-by-case basis. There must be some proof
that the medicine would either improve a person's quality of life or lengthen their
Risks of using investigational medicines
There are drawbacks to compassionate medicine use for you and for the medicine approval
process. One is that they may interfere with clinical trials if more people seek this
route rather than participating in trials. Although the NCI gathers some safety information
on the use of investigational medicines, it is not as helpful in getting FDA approval
of these medicines. You are not monitored as closely as you would be if you were part
of a clinical trial.
Other problems include:
Chances of getting no lasting benefit from the medicine
Undiscovered harmful side effects
Extra costs not covered by health insurance that may be linked to using the medicine
For more information
To learn more about investigational medicines and clinical trials, talk with your
healthcare provider about your options, the risks and side effects of the medicine
under consideration, and what the process is to apply to use the medicine. Clear communication
with the healthcare provider who treats you is important. Together, you can determine
whether a clinical trial or compassionate use is right for you.
Call NCI's Cancer Information Service at 800-4-CANCER (800-422-6237) or visit www.clinicaltrials.gov to search for clinical trials for your condition.
If you don't qualify for a clinical trial, your healthcare provider can ask the trial's
sponsor about requesting a single-patient exception. Your provider may also contact
the NCI or the company making the medicine to find out if expanded access programs